Journal article
Trials for Slowly Progressive Neurogenetic Diseases Need Surrogate Endpoints
Annals of neurology, Vol.93(5), pp.906-910
05/2023
DOI: 10.1002/ana.26633
PMCID: PMC10192108
PMID: 36891823
Appears in UI Libraries Support Open Access
Abstract
Heritable neurological disorders provide insights into disease mechanisms that permit development of novel therapeutic approaches including antisense oligonucleotides, RNA interference and gene replacement. Many neurogenetic diseases are rare and slowly progressive making it challenging to measure disease progression within short time frames. We share our experience developing clinical outcome assessments and disease biomarkers in the inherited peripheral neuropathies. We posit that carefully developed biomarkers from imaging, plasma or skin can predict meaningful progression in functional and patient reported outcome assessments such that clinical trials of less than two years will be feasible for these rare and ultra-rare disorders. This article is protected by copyright. All rights reserved.
Details
- Title: Subtitle
- Trials for Slowly Progressive Neurogenetic Diseases Need Surrogate Endpoints
- Creators
- Mary M Reilly - National Hospital for Neurology and NeurosurgeryDavid N Herrmann - University of RochesterDavide Pareyson - Fondazione IRCCS Istituto Neurologico Carlo BestaSteven S Scherer - University of PennsylvaniaRichard S Finkel - St. Jude Children's Research HospitalStephan Züchner - University of MiamiJoshua Burns - The University of SydneyMichael E Shy - Roy J. and Lucille A. Carver College of Medicine
- Resource Type
- Journal article
- Publication Details
- Annals of neurology, Vol.93(5), pp.906-910
- DOI
- 10.1002/ana.26633
- PMID
- 36891823
- PMCID
- PMC10192108
- NLM abbreviation
- Ann Neurol
- eISSN
- 1531-8249
- Publisher
- Wiley
- Grant note
- DOI: 10.13039/100005202, name: Muscular Dystrophy Association; DOI: 10.13039/100000065, name: National Institute of Neurological Disorders and Stroke, award: R01NS105755, U01NS109403, U54NS065712
- Language
- English
- Electronic publication date
- 03/09/2023
- Date published
- 05/2023
- Academic Unit
- Neurology; Molecular Physiology and Biophysics; Iowa Neuroscience Institute
- Record Identifier
- 9984375456102771
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